A French teenager’s sickle cell disease has been reversed using a pioneering treatment to change his DNA.
The world-first procedure at Necker Children’s Hospital in Paris offers hope to millions of people with the blood disorder. Scientists altered the genetic instructions in his bone marrow so it made healthy red blood cells.
So far, the therapy has worked for 15 months and the child is no longer on any medication. Sickle cell disease causes normally round red blood cells, which carry oxygen around the body, to become shaped like a sickle. These deformed cells can lock together to block the flow of blood around the body. This can cause intense pain, organ damage and can be fatal.
The teenager who received the treatment had so much internal damage he needed to have his spleen removed and his hips replaced. Read more